Article Text
Abstract
Objective: To evaluate the safety and efficacy of autologous stem cell transplantation (ASCT) for refractory juvenile idiopathic arthritis (JIA).
Design: Retrospective analysis of follow up data on 34 children with JIA who were treated with ASCT in nine different European transplant centres. Rheumatological evaluation employed a modified set of core criteria. Immunological reconstitution and infectious complications were monitored at three month intervals after transplantation.
Results: Clinical follow up ranged from 12 to 60 months. Eighteen of the 34 patients (53%) with a follow up of 12 to 60 months achieved complete drug-free remission. Seven of these patients had previously failed treatment with anti-TNF. Six of the 34 patients (18%) showed a partial response (ranging from 30% to 70% improvement) and seven (21%) were resistant to ASCT. Infectious complications were common. There were three cases of transplant related mortality (9%) and two of disease related mortality (6%).
Conclusions: ASCT in severely ill patients with JIA induces a drug-free remission of the disease and a profound increase in general wellbeing in a substantial proportion of patients, but the procedure carries a significant mortality risk. The following adjustments are proposed for future protocols: (1) elimination of total body irradiation from the conditioning regimen; (2) prophylactic administration of antiviral drugs and intravenous immunoglobulins until there is a normal CD4+ T cell count.
- ASCT, autologous stem cell transplantation
- ATG, anti-thymocyte immunoglobulin
- CHAQ, child health assessment questionnaire
- DMARD, disease modifying anti-rheumatic drug
- EBMT, European Group for Blood and Marrow Transplantation
- IAHS, infection associated haemophagocytic syndrome
- JIA, juvenile idiopathic arthritis
- NSAID, non-steroidal anti-inflammatory drug
- TBI, total body irradiation
- TNF, tumour necrosis factor
- VAS, visual analogue scale
- juvenile idiopathic arthritis
- autologous stem cell transplantation
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- ASCT, autologous stem cell transplantation
- ATG, anti-thymocyte immunoglobulin
- CHAQ, child health assessment questionnaire
- DMARD, disease modifying anti-rheumatic drug
- EBMT, European Group for Blood and Marrow Transplantation
- IAHS, infection associated haemophagocytic syndrome
- JIA, juvenile idiopathic arthritis
- NSAID, non-steroidal anti-inflammatory drug
- TBI, total body irradiation
- TNF, tumour necrosis factor
- VAS, visual analogue scale